The Safety and Tolerability of Engensis (VM202) in Patients With Charcot-Marie-Tooth Disease Subtype 1A (CMT1A)

• Register: ClinicalTrials.gov
• Last refreshed on: 9 June 2025
• Main ID: NCT05361031
• Date of registration: 31/03/2022
• Prospective Registration: No
• Primary sponsor: Helixmith Co., Ltd.
• Public title: The Safety and Tolerability of Engensis (VM202) in Patients With Charcot-Marie-Tooth Disease Subtype 1A (CMT1A)
• Scientific title: Single Center, Open Label, Repeat Intramuscular Administration, 270 Days, Phase I/2a Clinical Trial to Evaluate Safety, Tolerability of Investigational Product (Engensis: VM202) With Charcot-Marie-Tooth Disease Subtype 1A (CMT1A)
• Date of first enrolment: September 21, 2020
• Target sample size: 12
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT05361031
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

Korea, Republic of

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Males or females = 19 years of age and = 65 years of age

2. Patients with confirmed diagnosis of CMT1A by genetic testing

3. Patients with mild to moderate severity assessed by Charcot Marie Tooth Neuropathy
Score version 2 (CMTNS v2) with a score > 2 and = 20

4. Individuals with lower limb muscle weakness with minimum dorsiflexion or more

5. Individuals who voluntarily consented to participate in this study and signed the
IRB approved informed consent form after listening to a description on the
characteristics of this clinical study prior to all screening tests

6. Individuals who can comply with the requirements in the clinical study

7. In case of females of child bearing potential, those who test negative in a urine or
serum pregnancy test at screening

8. Individuals who practice medically approved contraceptive methods throughout the
clinical study

Exclusion Criteria:

1. Patients with significant respiratory, circulatory, renal, gastrointestinal,
hepatic, endocrine, hematologic, psychiatric disorders or other severe diseases, or
alcohol or drug addiction who may develop safety issues or cause confusion in the
interpretation of the clinical study results as determined by the principal
investigator

2. Patients with other neuromuscular diseases or neuropathy-inducing factors: Patients
with chronic alcohol addiction, undergoing anticancer chemotherapy, or taking
neurotoxic drugs

3. Patients diagnosed with diabetes

4. Patients diagnosed with inflammatory bowel disease

5. Patients with a history of stroke or cerebral ischemic attack within 12 months prior
to the screening date

6. Patients with a history of coronary artery disease, such as myocardial infarction
and unstable angina pectoris, within 12 months prior to the screening date

7. Morbidly obese patients with body mass index (BMI) = 37

8. Patients who underwent orthopedic surgery (corrective surgery for bone and ligament,
artificial joint implantation, osteosynthesis, osteotomy, arthroscopic surgery) in
the lower limbs within 6 months prior to the screening date

9. Patients who may be affected by the muscle strength measurement test due to ankle
contracture or surgery

10. Patients with uncontrolled hypertension (if systolic blood pressure is = 160 mmHg or
diastolic blood pressure is = 100 mmHg at screening)

11. Patients or patient's immediate family members (parents, siblings, offspring) with a
history of malignant tumors within the last 5 years prior to the screening date,
excluding basal cell carcinoma or squamous cell carcinoma that occurs on the skin
(if it is determined that there is no possibility of relapse after resection), or
with a family history of familial adenomatous polyposis (FAP) or hereditary
nonpolyposis colorectal cancer (HNPCC)

12. Patients who have not completed a national cancer screening program applicable to
their sex and age (if it cannot be confirmed that the relevant test was received at
a national cancer screening center or a recognized screening center)

However, if it is confirmed that the relevant test was received at a national cancer
screening center or a recognized screening center during the screening period, and
that the results were within normal range, the patients may participate in the
clinical study.

Common to males and females: If a patient is = 50 years of age, the results of a
colonoscopy within 5 years prior to the screening must be determined as being within
normal range, and if adenomatous polyps are evident, the results of a colonoscopy
within 1 year must be determined as being within normal range (inflammatory polyps
or hyperplastic polyps are included in the normal range). If a patient is = 40 years
of age, the results of a gastroscopy within 2 years prior to the screening must be
within normal range. If a patient is = 54 years of age and has a 30 pack-year
history of smoking or more, the results of a low-dose chest CT within 2 years prior
to the screening must be within normal range. In case of liver cancer, carriers of
hepatitis B or hepatitis C virus and patients with hepatic cirrhosis fall under the
exclusion criteria.

Females: For females = 40 years of age, normal range findings must be confirmed in a
mammogram within 2 years. For females = 20 years of age, normal range findings must
be confirmed in a Pap smear within 2 years.

13. Patients diagnosed with active pulmonary tuberculosis

14. Patients with HBV or HCV

15. Patients who test positive in human immunodeficiency virus (HIV) antibody test

16. Patients in an immunosuppressive state due to treatments such as immunosuppressants,
chemotherapy, and radiotherapy

17. Patients with a history of mental disease within 6 months prior to the screening
date, which may interfere with participation in the study

18. Patients who must take medications, that are known to have significant drug
interactions within 14 days after the first administration of the investigational
product or deemed unsuitable by the investigator's judgment

19. Individuals who participated in another clinical study within 6 months before the
time of screening

20. Individuals who have shown significant adverse events such as hypersensitivity
reactions to the investigational product

21. Pregnant or breastfeeding females

22. Other individuals determined ineligible by the principal investigator to participate
in the clinical study due to other reasons including clinical laboratory test
results


Age minimum: 19 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

CMT

Intervention(s)

Biological: Engensis (VM202)

Primary Outcome(s)

Safety of Intramuscular (IM) Injections of Engensis in Participants [Time Frame: Day 0 visit to the Day 270 visit]

Secondary Outcome(s)

Evaluation of Severity of Disease Changes Following Engensis Injections - Charcot Marie Tooth Neuropathy Score Version 2 [Time Frame: Day 0 and Day 270]

Change From Baseline in Nerve Conduction Velocity at Day 270 [Time Frame: Day 0 and Day 270]

Evaluation of Patient's Neurological Disability - Functional Disability Score [Time Frame: Day 0, 90, 180 and Day 270]

Evaluation of Patient's Walking Ability - Change From Baseline in the 10-Meter Walk Test [Time Frame: Day 0, 90, 180 and Day 270]

Changes in Fatty Infiltration Level of Lower Limb Muscles [Time Frame: Day 0 and Day 270]

Numbers of Participants With and Without Hepatocyte Growth Factor Antibody Generation in the Body at Baseline (Day 0) and Day 270. [Time Frame: Day 0 and Day 270]

Evaluation of the Activity Level of Patients With Peripheral Neuropathy Using the Overall Neuropathy Limitation Score Leg Scale [Time Frame: Day 0, 90, 180 and Day 270]

Secondary ID(s)

VMCMT-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 29/05/2025
• URL: https://clinicaltrials.gov/ct2/show/results/NCT05361031